Covering the whole development process for the global biotechnology industry

Bioprocessing begins upstream, most often with culturing of animal or microbial cells in a range of vessel types (such as bags or stirred tanks) using different controlled feeding, aerating, and process strategies.

Beginning with harvest of material from a bioreactor, downstream processing removes or reduces contaminants to acceptable levels through several steps that typically include centrifugation, filtration, and/or chromatographic technologies.

Drug products combine active pharmaceutical ingredients with excipients in a final formulation for delivery to patients in liquid or lyophilized (freeze-dried) packaged forms — with the latter requiring reconstitution in the clinical setting.

Many technologies are used to characterize biological products, manufacturing processes, and raw materials. The number of options and applications is growing every day — with quality by design (QbD) giving impetus to this expansion.

Even as it matures, the biopharmaceutical industry is still a highly entrepreneurial one. Partnerships of many kinds — from outsourcing to licensing agreements to consultancies — help companies navigate this increasingly global business environment.

Precision Bio $100m IPO to support allo CAR-T build-out

Precision Bio hopes the initial public offering (IPO) will advance its off-the-shelf CAR-T candidates, based on its ARCUS genome editing platform.

North Carolina-based Precision Bio has applied for a listing on the Nasdaq exchange. According to its Form S-1 filed with the SEC, the firm will use the anticipated $100 million (€88 million) raised in the IPO to advance and expand its clinical and preclinical development programs.

This includes plans to complete a Phase I/IIa clinical trial for its CD19 chimeric antigen receptor (CAR) T-cell candidate, PBCAR0191 for the treatment of acute lymphocytic leukemia and non-Hodgkin lymphoma, and expand the preclinical development of its in vivo gene correction platform.

Image: iStock/nelsonpeng

The candidates are based on its proprietary genome editing platform, ARCUS, which uses sequence-specific DNA-cutting enzymes – or nucleases – designed to perform modifications in the DNA of living cells and organism.

“ARCUS is not a CRISPR/Cas9 technology,” the firm says in the filing. “ARCUS is a collection of protein engineering methods that we developed specifically to re-program the DNA recognition properties of the natural genome editing enzyme, I-CreI. In nature, I-CreI is an endonuclease found in the genome of the algae Chlamydomonas reinhardtii, which evolved for the purpose of carrying out a complex gene insertion edit.”

The firm believes the platform has several attributes for genome editing applications, including high specificity, high efficiency, ease of delivery, the type of DNA cut and the programmability.

“We have used the qualities of ARCUS to develop a one-step cell engineering process for allogeneic CAR T-cells that is designed to rapidly yield a consistent cell product at a significantly lower cost than autologous CAR T-cell therapies,” the firm says.

“Due to our one-step editing method and the decision early in the development of our allogeneic CAR-T immunotherapy platform to invest in process development, we have scaled our manufacturing process and are currently producing allogeneic CAR T-cells at large scale in accordance with good manufacturing practice, or GMP.”

The net proceeds of the IPO will also be used to fund the build-out of a cGMP-compliant manufacturing facility in Durham, North Carolina to support the candidates.

“We have leased approximately 17,300 square feet of space for our manufacturing facility at a location approximately seven miles from our headquarters in Durham, North Carolina, at which we intend to establish and equip a manufacturing facility compliant with cGMP,” the prospectus says.